Krystal Biotech, Inc. is a clinical-stage biotechnology company focused on developing redosable gene therapies to address severe orphan diseases across the United States. Its most advanced therapeutic candidate, beremagene geperpavec (B-VEC), is currently undergoing Phase III clinical evaluation for the treatment of dystrophic epidermolysis bullosa. The company's pipeline also includes several other promising programs: KB105 is in Phase I/II clinical trials for patients suffering from deficient autosomal recessive congenital ichthyosis; KB301 is also in Phase I/II development, aiming to ameliorate wrinkles and other signs of aging or damaged skin. Additionally, Krystal Biotech is conducting preclinical research on KB407 for cystic fibrosis and KB104 for Netherton syndrome. In its earlier discovery phase, the company is exploring candidates such as KB5xx for chronic skin conditions and multiple KB3xx programs targeting aesthetic dermatological concerns. Krystal Biotech, Inc. was established in 2015 and is based in Pittsburgh, Pennsylvania.
